DTx-1252 is a progressive neuromuscular autosomal-dominant disease. Credit: OlgaReukova /Shutterstock.com.

The US Food Drug Administration (FDA) has granted orphan drug designation to DTx Pharma’s investigational DTx-1252 for the treatment of Charcot-Marie-Tooth disease Type 1A (CMT1A).

DTx-1252 is a fatty acid ligand conjugated oligonucleotides (FALCON) small interfering RNA (siRNA) therapeutic which represses the PMP22 gene in Schwann cells.

Related Company Profiles

View all

It induces remyelination of axons to normal levels, increases muscle coordination, agility, mass, grip and strength, and improves electrophysiological measurements.

DTx Pharma co-founder and CEO Artie Suckow stated: “Patients living with CMT1A are in urgent need of treatment and this represents a significant step forward for the patients and the DTx team as we advance DTx-1252 toward the clinic.

“DTx-1252 targets the underlying genetic lesion of the disease and leverages our FALCON platform to unlock the promise of RNAi therapeutics.

See Also:

“We look forward to the continued development of DTx-1252.”

How well do you really know your competitors?

Access the most comprehensive Company Profiles on the market, powered by GlobalData. Save hours of research. Gain competitive edge.

Company Profile – free sample

Thank you!

Your download email will arrive shortly

Not ready to buy yet? Download a free sample

We are confident about the unique quality of our Company Profiles. However, we want you to make the most beneficial decision for your business, so we offer a free sample that you can download by submitting the below form

By GlobalData
Visit our Privacy Policy for more information about our services, how we may use, process and share your personal data, including information of your rights in respect of your personal data and how you can unsubscribe from future marketing communications. Our services are intended for corporate subscribers and you warrant that the email address submitted is your corporate email address.

FALCON siRNAs can be manufactured at low cost and delivered intrathecally [via the spine], intravenously and subcutaneously.

CMT1A is a progressive neuromuscular autosomal-dominant disease that leads to life-long loss of muscle function, as well as disability. It significantly impacts patients’ lives, causing loss of mobility, progressive muscle wasting and neuropathic pain.

An estimated 150,000 patients in Europe and the US are living with CMT1A.

Editorial content is independently produced and follows the highest standards of journalistic integrity. Topic sponsors are not involved in the creation of editorial content.